Molecular Therapy-Methods & Clinical Development

Article	Citations
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy	99
Thank you to our 2024 reviewers	71
Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP	65
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice	64
Exploring human plasma proteomic variations in mucolipidosis type IV	64
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy	59
Nonclinical pharmacokinetics and biodistribution of VSV-GP using methods to decouple input drug disposition and viral replication	58
Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8	57
Genetic surgery for a cystic fibrosis-causing splicing mutation	56
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	55
Delivery of nVEGFi using AAV8 for the treatment of neovascular age-related macular degeneration	54
Molecular earplugs to protect the inner ear	53
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model	51
Novel TCR-like CAR-T cells targeting an HLA∗0201-restricted SSX2 epitope display strong activity against acute myeloid leukemia	49
Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates	45
First use of adeno-associated viruses in the human inner ear	43
RNA-seq analysis of the human surfactant air-liquid interface culture reveals alveolar type II cell-like transcriptome	43
Differential T cell immune responses to deamidated adeno-associated virus vector	43
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy	41
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration	39
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids	38
Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia	38
Safety and efficiency modifications of SIV-based integrase-defective lentiviral vectors for immunization	38
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	37
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy	36

AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear	35
Synergy between Lactobacillus murinus and anti-PcrV antibody delivered in the airways to boost protection against Pseudomonas aeruginosa	34
Modulation of AAV transduction and integration targeting by topoisomerase poisons	34
Optimization of anti-CD19 CAR T cell production for treatment of patients with chronic lymphocytic leukemia	33
An investigation of the immune epitopes of adeno-associated virus capsid-derived peptides among hemophilia patients	32
Efficacy and muscle safety assessment of fukutin-related protein gene therapy	31
An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development	30
The unknown impact of conditioning on HSC engraftment and clonal dynamics	30
Automated manufacture of ΔNPM1 TCR-engineered T cells for AML therapy	30
Temporal insights into molecular and cellular responses during rAAV production in HEK293T cells	29
Applying a clinical lens to animal models of CAR-T cell therapies	29
Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay	29
Deconvolution of spatial sequencing provides accurate characterization of hESC-derived DA transplants in vivo	29
A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies	29
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy	29
Determining recombinant AAV capsid extracellular and intracellular biodistribution by dual radioisotope labeling	29
Evidence generation and reproducibility in cell and gene therapy research: A call to action	29
Efficacy of HSV-TK/GCV system suicide gene therapy using SHED expressing modified HSV-TK against lung cancer brain metastases	29
Defining the optimal dose and therapeutic window in SMA with respiratory distress type I model mice, FVB/NJ-Ighmpb2	28
Inducible caspase 9-mediated suicide gene therapy using AAV6 vectors in a murine model of breast cancer	28
The longitudinal kinetics of AAV5 vector integration profiles and evaluation of clonal expansion in mice	28
Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A	28
A combinatorial CRISPR-Cas12a attack on HIV DNA	27
Development of novel lipoplex formulation methodologies to improve large-scale transient transfection for lentiviral vector manufacture	27
Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy	26
A lentiviral vector B cell gene therapy platform for the delivery of the anti-HIV-1 eCD4-Ig-knob-in-hole-reversed immunoadhesin	26
Nonviral delivery of nCas9 for “safe harbor” integration to treat MPS IVA	24
CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice	24
Transcriptomic analysis reveals optimal cytokine combinations for SARS-CoV-2-specific T cell therapy products	24
Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors	24
Suppression of toxic transgene expression by optimized artificial miRNAs increases AAV vector yields in HEK-293 cells	24
A linear DNA encoding the SARS-CoV-2 receptor binding domain elicits potent immune response and neutralizing antibodies in domestic cats	23
In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans	23
Confirmatory detection of neutralizing antibodies to AAV gene therapy using a cell-based transduction inhibition assay	23
Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice	23
A novel approach to quantitate biodistribution and transduction of adeno-associated virus gene therapy using radiolabeled AAV vectors in mice	23
Monitoring CAR T cell generation with a CD8-targeted lentiviral vector by single-cell transcriptomics	23
Co-transducing B7H3 CAR-NK cells with the DNR preserves their cytolytic function against GBM in the presence of exogenous TGF-β	23
Lipid nanoparticles: Composition, formulation, and application	23
Emerging therapeutic potential of adeno-associated virus-mediated gene therapy in liver fibrosis	23
In vitro and in vivo expansion of CD33/HBG promoter-edited HSPCs with Mylotarg	22
Development of circular AAV cargos for targeted seamless insertion with large serine integrases	22
Ionizable lipid nanoparticles with functionalized PEG-lipids increase retention in the tumor microenvironment	22
Bringing base editing to the clinic: The next generation of genome editors	21
Manufacturing DNA in E. coli yields higher-fidelity DNA than in vitro enzymatic synthesis	21
AAV8BP2 and AAV8 transduce the mammalian cochlear lateral wall and endolymphatic sac with high efficiency	21
Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications	21
Depletion of high-content CD14+ cells from apheresis products is critical for successful transduction and expansion of CAR T cells during large-scale cGMP manufacturing	20
Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model	20
Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models	20

Comparison of analytical techniques to quantitate the capsid content of adeno-associated viral vectors	20
Cell-penetrating peptides enhance the transduction of adeno-associated virus serotype 9 in the central nervous system	20
Modulation of the pharmacokinetics of soluble ACE2 decoy receptors through glycosylation	20
Lentiviral and adeno-associated vectors efficiently transduce mouse T lymphocytes when targeted to murine CD8	20
Characterization and modulation of anti-αβTCR antibodies and their respective binding sites at the βTCR chain to enrich engineered T cells	20
Linker-specific monoclonal antibodies present a simple and reliable detection method for scFv-based CAR NK cells	20
Extracellular vesicles ameliorates sleep deprivation induced anxiety-like behavior and cognitive impairment in mice	20
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques	20
Research progress of mosquito-borne virus mRNA vaccines	20
Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity	20
Simultaneous engineering of natural killer cells for CAR transgenesis and CRISPR-Cas9 knockout using retroviral particles	19
Toward lentiviral vectors for antiangiogenic ocular gene therapy	19
Optimizing AAV2/6 microglial targeting identified enhanced efficiency in the photoreceptor degenerative environment	19
Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors	19
Antigen modifications improve nucleoside-modified mRNA-based influenza virus vaccines in mice	19
Assessment of genome packaging in AAVs using Orbitrap-based charge-detection mass spectrometry	19
Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6	19
CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease	19
Universal ddPCR-based assay for the determination of lentivirus infectious titer and lenti-modified cell vector copy number	19
Non-canonical capsid engineering highlights new possibilities for AAV vectorology	18
Efficient and sustained FOXP3 locus editing in hematopoietic stem cells as a therapeutic approach for IPEX syndrome	18
Toward effective hematopoietic stem cell gene therapies: Optimized conditioning regimen and stem cell source in harmony	18
Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome	18
Starburst amacrine cells amplify optogenetic visual restoration through gap junctions	18
Human amnionic progenitor cell secretome mitigates the consequence of traumatic optic neuropathy in a mouse model	18
Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device	18
Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers	17
Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease	17
Transduction characteristics of alternative adeno-associated virus serotypes in the cat brain by intracisternal delivery	17
mRNA-LNP vaccine strategies: Effects of adjuvants on non-parenchymal liver cells and tolerance	17
Reversal of neuroinflammation in novel GS model mice by single i.c.v. administration of CHO-derived rhCTSA precursor protein	17
VikAD, a Vika site-specific recombinase-based system for efficient and scalable helper-dependent adenovirus production	17
It’s all about location: Targeting the right spot for Wiskott-Aldrich syndrome	17
Brain-targeted ex vivo lentiviral gene therapy: Implications for MPS and beyond	17
Assessment of adeno-associated virus purity by capillary electrophoresis-based western	17
Improving cell-specific recombination using AAV vectors in the murine CNS by capsid and expression cassette optimization	17
Identifying MAGE-A4-positive tumors for TCR T cell therapies in HLA-A∗02-eligible patients	17
Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model	17
Optimization of hypo-alloimmunogenic multispecific CAR-T and SARS-CoV-2-specific T cells for off-the-shelf adoptive cell therapy	17
Rationally engineered novel AAV capsids for intra-articular gene delivery	16
Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid	16
Immunosuppression reduces rAAV2.5T neutralizing antibodies that limit efficacy following repeat dosing to ferret lungs	16
Low endotoxin E. coli strain-derived plasmids reduce rAAV vector-mediated immune responses both in vitro and in vivo	16
Induction of antigen-specific tolerance by hepatic AAV immunotherapy regardless of T cell epitope usage or mouse strain background	16
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies	16
Lentiviral vector mediated gene therapy for type I Dent disease ameliorates Dent disease-like phenotypes for three months in ClC-5 null mice	16
Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity	16
Necessity of strengthening the current clinical regulatory for companion diagnostics: An institutional comparison of the FDA, EMA, and MFDS	16
Targeted biallelic integration of an inducible Caspase 9 suicide gene in iPSCs for safer therapies	16
Engineered mesenchymal stromal cell therapy during human lung ex vivo lung perfusion is compromised by acidic lung microenvironment	16
A flexible, thermostable nanostructured lipid carrier platform for RNA vaccine delivery	16
Atelocollagen supports three-dimensional culture of human induced pluripotent stem cells	16
Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2	15
Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease	15
Efficient generation of liver sinusoidal endothelial-like cells secreting coagulation factor VIII from human induced pluripotent stem cells	15
Gene therapy restores adipose tissue and metabolic health in a pre-clinical mouse model of lipodystrophy	15
PCR-based analytics of gene therapies using adeno-associated virus vectors: Considerations for cGMP method development	15
Continuous manufacturing of lentiviral vectors using a stable producer cell line in a fixed-bed bioreactor	15
In vivo targeting of a variant causing vanishing white matter using CRISPR/Cas9	15
FAP-CAR-T cells reduce dystrophic muscle fibrosis, improving Adeno-Associated Virus gene transfer efficacy	15
A randomized, double-blind phase 2b trial to evaluate efficacy of ChAd63-KH for treatment of post kala-azar dermal leishmaniasis	15
Characterization and effective expansion of CD4−CD8− TCRαβ+ T cells from individuals living with type 1 diabetes	15
Packaging cells for lentiviral vectors generated using the cumate and coumermycin gene induction systems and nanowell single-cell cloning	15
Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells	15
A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR	15
Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations	15
Gene therapy for Friedreich ataxia: Too much, too little, or just right?	15
Deciphering key parameters enhancing lentiviral vector producer cells yields: Vector components copy number and expression	15
Adeno-associated virus serotype 9 antibodies in neonates and young children: Seroprevalence and kinetics	14
Engineering a highly durable adeno-associated virus receptor for analytical applications	14
Engineering new metabolic pathways in isolated cells for the degradation of guanidinoacetic acid and simultaneous production of creatine	14
Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice	14
Efficient autocrine and paracrine signaling explain the osteogenic superiority of transgenic BMP-2 over rhBMP-2	14
Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy	14
A positive take on negative selection for CAR-T manufacturing	14
Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease	14
Persistent tailoring of MSC activation through genetic priming	14
Perspectives of the Friedreich ataxia community on gene therapy clinical trials	14
DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34+ cell clonogenic potential	14
A clinically viable approach to restoring visual function using optogenetic gene therapy	14

Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection	14
Peptide-encoding gene transfer to modulate intracellular protein-protein interactions	14
Follistatin-like 1 promotes proliferation of matured human hypoxic iPSC-cardiomyocytes and is secreted by cardiac fibroblasts	13
A rescue Fanconi anemia humanized mouse model with endogenous FA mutation and high human hematopoietic stem cell chimerism	13
Insights in AAV-mediated antigen-specific immunity and a strategy for AAV vaccine dose reduction through AAV-extracellular vesicle association	13
AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life	13
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography	13
CRISPR-Cas9-mediated genome editing delivered by a single AAV9 vector inhibits HSV-1 reactivation in a latent rabbit keratitis model	13
Impact of an autophagy-inducing peptide on immunogenicity and protection efficacy of an adenovirus-vectored SARS-CoV-2 vaccine	13
Preclinical safety assessment of MV-s-NAP, a novel oncolytic measles virus strain armed with an H. pylori immunostimulatory bacterial transgene	13
Optimized pharmacological control over the AAV-Gene-Switch vector for regulable gene therapy	13
Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette	13
AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency	13
Production of recombinant adeno-associated virus 5 using a novel self-attenuating adenovirus production platform	13
From Puppies to adults: In vivo editing of hepatocytes in a canine model of glycogen storage disease type Ia	13
mRNA-based therapy proves superior to the standard of care for treating hereditary tyrosinemia 1 in a mouse model	12
An autonucleolytic suspension HEK293F host cell line for high-titer serum-free AAV5 and AAV9 production with reduced levels of DNA impurity	12
An automated and high-throughput approach for enhanced precision of adenoviral titering	12
Can mitochondria brown the lower-limb adipocytes?	12
Exogenous expression of ATP8, a mitochondrial encoded protein, from the nucleus in vivo	12
Evaluation of efficacy and safety of AAV8-ΔC4ATP7B gene therapy in a mutant mouse model of Wilson’s disease	12
Measurement solutions and standards for advanced therapy	12
Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice	12
Self-amplifying mRNA bicistronic influenza vaccines raise cross-reactive immune responses in mice and prevent infection in ferrets	12
Lipid nanoparticle encapsulation of a Delta spike-CD40L DNA vaccine improves effectiveness against Omicron challenge in Syrian hamsters	11
Intravenous esketamine in pediatric Rett syndrome: An open-label, early phase 1 pilot study	11
miR-223-3p and miR-24-3p as novel serum-based biomarkers for myotonic dystrophy type 1	11
Clinical holds for cell and gene therapy trials: Risks, impact, and lessons learned	11
Case report of selumetinib as a novel therapy in a neurofibromatosis type 2-associated ependymoma	11
Comparing molecular and computational approaches for detecting viral integration of AAV gene therapy constructs	11
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys	11
Metabolic priming of GD2 TRAC-CAR T cells during manufacturing promotes memory phenotypes while enhancing persistence	11
CRISPR-Cas9-based non-viral gene editing therapy for topical treatment of recessive dystrophic epidermolysis bullosa	11
Strong ubiquitous micro-promoters for recombinant adeno-associated viral vectors	11
Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy	11
High throughput screening of novel AAV capsids identifies variants for transduction of adult NSCs within the subventricular zone	11
Self-amplifying mRNA SARS-CoV-2 vaccines raise cross-reactive immune response to variants and prevent infection in animal models	11
B cell focused transient immune suppression protocol for efficient AAV readministration to the liver	11
AAV-Mediated DUX4 Knockdown in Facioscapulohumeral Muscular Dystrophy: Therapeutic Benefits and Challenges for Long-Term Efficacy	11
Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency	11
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II	11